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UniQure must conduct another study to prove that its gene therapy “actually helps people with Huntington’s disease,” a senior U.S. Food and Drug Administration official said Thursday in a call with reporters.
The official, who requested anonymity before discussing sensitive information, confirmed that the agency had asked the company to conduct a placebo-controlled trial of its treatment, which is delivered directly to the brain. UniQure said this type of study was unethical because it would require placing people under general anesthesia for hours, a characterization disputed by the official.
“So what’s really going on? UniQure is the latest company to develop a failed treatment for Huntington’s patients,” the official said. “They probably recognize or understand on a deeper level that their trial failed years ago, and instead of doing the right thing and conducting the right clinical study, UniQure is doing a distorted or manipulated comparison in the mind of the FDA.”
The comments mark the latest development in a messy public spat between UniQure and the FDA, and as the agency comes under fire for a number of recent rejections of drug approval applications, including in some cases where companies have accused it of rolling back previous guidance. FDA Commissioner Marty Makary, in an interview with CNBC’s Becky Quick last week, apparently criticized UniQure’s gene therapy for Huntington’s disease. Makary did not name UniQure but described its treatment.
UniQure later accused the FDA of changing its position that the company’s clinical trial data would be sufficient to seek approval. The UniQure study used an external database to measure how well Huntington’s disease patients might decline without treatment, called an external control. UniQure said it would not be possible to conduct a true randomized, double-blind, placebo-controlled study, considered the gold standard, because it would not be ethical to force people to undergo hours-long fake brain surgery.
The FDA official said the agency “has never accepted this distorted comparison” and that the FDA “never gives such assurances.” Instead, the “FDA will always say, ‘Well, we need to see the data when we get it.’ » »
UniQure did not immediately comment.
The company’s shares rose more than 10% on Thursday and were down 58% this year as of Thursday afternoon.